Complete preclinical dataset to be presented at Society for Neuroscience annual meeting, November 18, 2014
CARLSBAD, CA. - (Marketwired – October 2, 2014), International Stem Cell Corporation (OTCQB: ISCO), a California-based biotechnology company developing novel stem cell based therapies and biomedical products today provided an update on the status of Parkinson’s disease preclinical program. The program continues to progress on the plan developed with input from the FDA. To date no adverse events or pathological reactions have been observed in any of the completed preclinical pharmacology/toxicology studies. Results from these studies will form the basis of the regulatory submission requesting authorization to administer the drug in humans that ISCO expects to submit prior to the end of the year.
“We continue to successfully carry out preclinical studies using our proprietary neural stem cells in well-validated animal models of Parkinson’s disease. The results so far demonstrate that the cells engraft, integrate and differentiate into functional dopaminergic neurons to recover the nigrostriatal system in the host brain. Importantly, no evidence of teratoma formation or ectopic tissue has been observed to date,” said Dr. Ruslan Semechkin, ISCO’s Chief Scientific Officer.“Once the few remaining steps in the preclinical development program are complete we will be submitting our investigational drug application and, with the regulatory agency’s approval, begin the Phase I/II clinical trial. The complete preclinical dataset will be presented at the Society for Neuroscience annual meeting on November 18, 2014”
The results of the latest studies compliment the acute toxicity study announced in June of this year, where healthy rodents received escalating doses of human parthenogenetic neural stem cell transplants to assess the immediate toxicological effects if any and identify a safe upper limit on the number of cells that can be safely transplanted. Behavioral observations along with necropsy data showed that the transplanted cells were in fact well tolerated, even at doses well above the anticipated clinical dose.
ISCO’s preclinical program involves a number of animal studies designed to assess the safety and tolerability of our novel cell therapy as well as providing a guide to the range of doses that can be used in the first clinical trial in people with Parkinson’s disease (PD). The PD program uses human parthenogenetic neural stem cells, a novel therapeutic cellular product derived from the company's proprietary human pluripotent stem cells. Neural stem cells are self-renewing multipotent cells that are precursors for the main cell types of the central nervous system. The ability of ISC-hpNSC to differentiate into dopaminergic neurons and express brain-protecting neurotrophic factors offers a new opportunity for the treatment of Parkinson's disease.
About Parkinson’s disease
According to the Parkinson’s Disease Foundation, an estimated seven to 10 million people worldwide live with Parkinson’s disease, with as many as one million of those in the United States alone, more than the combined total of people diagnosed with multiple sclerosis, muscular dystrophy, and Lou Gehrig’s disease. PD is a progressive neurodegenerative disorder resulting from the gradual loss of certain neurons responsible for producing dopamine, and is characterized by symptoms including tremors at rest, rigidity and impaired movement.
The standard of care for the treatment of PD symptoms is oral levodopa (L-dopa). Oral dosing of L-dopa is associated with wide variability in the timing and amount of L-dopa absorption into the bloodstream, leading to the unreliable control of symptoms resulting in the emergence of OFF episodes. These OFF episodes, which increase in frequency and severity during the course of the disease, are experienced by a majority of PD patients and are considered one of the greatest unmet medical needs facing PD patients.
About International Stem Cell Corporation
International Stem Cell Corporation is focused on the therapeutic applications of human parthenogenetic stem cells (hpSCs) and the development and commercialization of cell-based research and cosmetic products. ISCO's core technology, parthenogenesis, results in the creation of pluripotent human stem cells from unfertilized oocytes (eggs). hpSCs avoid ethical issues associated with the use or destruction of viable human embryos. ISCO scientists have created the first parthenogenetic, homozygous stem cell line that can be a source of therapeutic cells for hundreds of millions of individuals of differing genders, ages and racial background with minimal immune rejection after transplantation. hpSCs offer the potential to create the first true stem cell bank, UniStemCell™. ISCO also produces and markets specialized cells and growth media for therapeutic research worldwide through its subsidiary Lifeline Cell Technology (www.lifelinecelltech.com), and stem cell-based skin care products through its subsidiary Lifeline Skin Care (www.lifelineskincare.com). More information is available at www.internationalstemcell.com or follow us on Twitter @intlstemcell.
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Statements pertaining to anticipated developments, the expected timing and results of preclinical studies and subsequent regulatory filings, the potential benefits of research programs and products, and other opportunities for the company and its subsidiaries, along with other statements about the future expectations, beliefs, goals, plans, or prospects expressed by management constitute forward-looking statements. Any statements that are not historical fact (including, but not limited to statements that contain words such as "will," "believes," "plans," "anticipates," "expects," "estimates,") should also be considered to be forward-looking statements. Forward-looking statements involve risks and uncertainties, including, without limitation, risks inherent in the development and/or commercialization of potential products, regulatory approvals, need and ability to obtain future capital, application of capital resources among competing uses, and maintenance of intellectual property rights. Actual results may differ materially from the results anticipated in these forward-looking statements and as such should be evaluated together with the many uncertainties that affect the company's business, particularly those mentioned in the cautionary statements found in the company's Securities and Exchange Commission filings. The company disclaims any intent or obligation to update forward-looking statements.
International Stem Cell Corporation
Dr. Simon Craw, Executive Vice President